Project description
A novel drug to restore cognitive ability in Down syndrome patients
Down syndrome is a genetic disorder caused by the presence of an extra chromosome 21. People with Down syndrome have mild to moderate cognitive and learning deficits as well as certain physical features. Emerging evidence indicates that cognitive decline is due to the over-expression of the DYRK1A kinase in the brain. In neurons, the DYRK1A protein is involved in the formation and maturation of dendrite extensions which transmit nerve impulses. Funded by the European Innovation Council, the DOWN-AUTONOMY project will develop an inhibitor against DYRK1A protein as a novel therapeutic to reduce symptoms in Down syndrome patients.
Objective
All people with Down Syndrome (DS) suffer from a certain level of impaired learning and memory (cognitive disorders) from childhood onwards, greatly impacting their autonomy and societal integration. This is becoming an increasingly urgent issue as people with DS are more often outliving their care takers, yet an adequate treatment has not yet been developed.
Studies have revealed that the cognitive decline in people with DS is associated with overexpression of the DYRK1A protein kinase in the brain. Perha Pharmaceuticals has developed a novel therapeutic compound, Leucettinib-21 (LCTB-21), which acts as a DYRK1A inhibitor that can reduce cognitive decline in children with DS. Ultimately, this will increase the autonomy of people with DS resulting in better societal integration.
Perha Pharmaceuticals aims to outlicense LCTB-21 to a large pharmaceutical partner (e.g. Otsuka Pharmaceuticals, Intra-Cellular Therapies) following a successful phase 2a clinical trial.
Fields of science (EuroSciVoc)
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CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques.
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Programme(s)
- HORIZON.3.1 - The European Innovation Council (EIC) Main Programme
Funding Scheme
HORIZON-EIC-ACC-BF - HORIZON EIC Accelerator Blended FinanceCoordinator
29680 Roscoff
France
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.